Search Clinical Trials
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National Collaborative to Improve Care of Children With Complex Congenital Heart Disease
Children's Hospital Medical Center, Cincinnati
Hypoplastic Left Heart Syndrome (HLHS)
The purpose of this initiative is to improve care and outcomes for infants with HLHS by
expanding the NPC-QIC national registry to gather clinical care process, outcome, and
developmental data on infants with HLHS between diagnosis and 12 months of age, by improving
the use... expand
The purpose of this initiative is to improve care and outcomes for infants with HLHS by expanding the NPC-QIC national registry to gather clinical care process, outcome, and developmental data on infants with HLHS between diagnosis and 12 months of age, by improving the use of standards into everyday practice across pediatric cardiology centers, and by engaging parents as partners in the process. Type: Observational [Patient Registry] Start Date: May 2016 |
Natural History and Biospecimen Acquisition for Children and Adults With Rare Solid Tumors
National Cancer Institute (NCI)
Malignant Solid Tumors
Other Neoplasms Solid Tumors
Pediatric Solid Tumor
Refractory Solid Tumors
Solid Tumor
Background:
Approximately 150 cases of cancer per one million per year are considered rare cancers. While
all tumors originate from genetic changes, a small percentage of these tumors are familial.
Researchers want to study these changes in biological samples from people... expand
Background: Approximately 150 cases of cancer per one million per year are considered rare cancers. While all tumors originate from genetic changes, a small percentage of these tumors are familial. Researchers want to study these changes in biological samples from people with rare tumors in order to learn more about how these tumors develop. The information obtained from this study may lead to improved screening, preventive guidelines, and treatments. Objective: To better understand rare cancers and hereditary cancer syndromes. Eligibility: People who have a rare tumor, a family history of a rare tumor, a hereditary cancer syndrome, or a mutation that leads to rare tumors. Design: Participants will be screened with questions about their medical history and/or that of their family members. They will give a saliva sample. Participants who have a tumor will have their medical records and tests reviewed. They will answer questions about their wellbeing and needs. They may provide a tumor tissue sample. Participants may also have: - Physical exam - Clinical photography - Blood, urine, saliva, and stool samples taken - Consultation with specialists - A scan that produces a picture of the body. Either one that uses a small amount of radiation, or one that uses a magnetic field. - Genetic testing/genetic counseling. Participants will be contacted once a year. They will answer updated questions about their medical and family history. Participants will be asked to contact the study team if there are changes in their tumors. Participants may be invited to join focus groups for people with the same diagnosis of rare tumors. Participants may be invited to participate in other NIH protocols. **************************************** **************************************** RARE TUMOR LIST: 1. Acinar cell carcinoma of the pancreas 2. Adamantinoma 3. Adenosqaumous carcinoma of the pancreas 4. Adrenocortical carcinoma 5. Alveolar soft part sarcoma 6. Anaplastic Thyroid Cancer 7. Angiosarcoma 8. Atypical Teratoid Rhabdoid Tumor/MRT 9. Carcinoid 10. Carcinoma of Unknown Primary 11. Chondrosarcoma 12. Chondromyxoid fibroma 13. Chordoma 14. Clear cell renal carcinoma 15. Clear Cell Sarcoma 16. Clear cell sarcoma of kidney 17. Conventional chordoma 18. Dedifferentiated chordoma 19. Desmoid 20. Desmoplastic small round cell tumor 21. Epithelioid hemangioendothelioma 22. Esthenioneuroblastoma 23. Ewing Sarcoma 24. Fibrolamellar carcinoma 25. Fusion negative rhabdomyosarcoma 26. Fusion positive renal cell carcinoma 27. Fusion positive rhabdomyosarcoma 28. Gastro-enteropancreatic neuroendocrine tumor 29. Hepatoblastoma 30. Hereditary Diffuse Gastric Cancer 31. Inflammatory myofibroblastic tumor 32. Kaposiform hemangioendothelioma 33. Malignant ectomesenchymal tumor 34. Malignant peripheral nerve sheath tumor 35. Malignant triton tumor 36. Medullary thyroid cancer 37. Mixed acinar adenocarcinoma 38. Mixed acinar neuroendocrine carcinoma 39. Myxoid Liposarcoma 40. Neuroblastoma 41. Neuroendocrine tumors 42. NUT midline carcinoma 43. Osteosarcoma 44. Pancreas ductal adenocarcinoma with squamous features 45. Pancreatic acinar cell carcinoma 46. Papillary renal cell carcinoma 47. Paraganglioma 48. Parosteal Osteosarcoma 49. Periosteal Osteosarcoma 50. Peripheral nerve sheath tumor 51. Peripheral primitive neuroectodermal tumor 52. Pheochromocytoma 53. Pituitary cancer 54. Poorly differentiated chordoma 55. Renal medullary carcinoma 56. Rhabdomyosarcoma 57. Round cell Liposarcoma 58. Schwannoma 59. Sclerosing Epithelioid Fibrosarcoma 60. SDH deficient GIST 61. SMARCB1 deficient tumors 62. SMARCA4 deficient tumors 63. Synovial sarcoma 64. Undifferentiated Sarcoma **************************************** **************************************** Type: Observational Start Date: Jan 2019 |
Clinical and Genetic Evaluation of Individuals With Undiagnosed Disorders Through the Undiagnosed Diseases...
National Human Genome Research Institute (NHGRI)
Genetic Disease
Without an explanation for severe and sometimes life-threatening symptoms, patients and their
families are left in a state of unknown. Many individuals find themselves being passed from
physician to physician, undergoing countless and often repetitive tests in the hopes of... expand
Without an explanation for severe and sometimes life-threatening symptoms, patients and their families are left in a state of unknown. Many individuals find themselves being passed from physician to physician, undergoing countless and often repetitive tests in the hopes of finding answers and insight about what the future may hold. This long and arduous journey to find a diagnosis does not end for many patients- the Office of Rare Diseases Research (ORDR) notes that 6% of individuals seeking their assistance have an undiagnosed disorder. In 2008, the National Institutes of Health (NIH) Undiagnosed Diseases Program (UDP) was established with the goal of providing care and answers for these individuals with mysterious conditions who have long eluded diagnosis. The NIH UDP is a joint venture of the NIH ORDR, the National Human Genome Research Institute Intramural Research Program (NHGRI-IRP), and the NIH Clinical Research Center (CRC) (1-3). The goals of the NIH UDP are to: (1) provide answers for patients with undiagnosed diseases; (2) generate new knowledge about disease mechanisms; (3) assess the application of new approaches to phenotyping and the use of genomic technologies; and (4) identify potential therapeutic targets, if possible. To date, the UDP has evaluated 3300 medical records and admitted 750 individuals with rare and undiagnosed conditions to the NIH Clinical Center. The NIH UDP has identified more than 70 rare disease diagnoses and several new conditions. The success of the NIH UDP prompted the NIH Common Fund to support the establishment of a network of medical research centers, the Undiagnosed Diseases Network (UDN), for fiscal years 2013-2020. The clinical sites will perform extensive phenotyping, genetic analyses, and functional studies of potential disease-causing variants. The testing performed on patients involves medically indicated studies intended to help reach a diagnosis, as well as research investigations that include a skin biopsy, blood draws, and DNA analysis. In addition, the UDN will further the goals of the UDP by permitting the sharing of personally identifiable phenotypic and genotypic information within the network. By sharing participant information and encouraging collaboration, the UDN hopes to improve the understanding of rare conditions and advance the diagnostic process and care for individuals with undiagnosed diseases. Type: Observational Start Date: Sep 2015 |
A Master Protocol (LY900023) That Includes Several Clinical Trials of Drugs for Children and Young Adults...
Eli Lilly and Company
Neoplasms
Child
Adolescent
The main purpose of the master is to help the research sites and sponsor carry out several
clinical trials more efficiently by providing a common research protocol. Individual clinical
trials under this master protocol define drug/disease-specific research goals and activities... expand
The main purpose of the master is to help the research sites and sponsor carry out several clinical trials more efficiently by providing a common research protocol. Individual clinical trials under this master protocol define drug/disease-specific research goals and activities to test them. New studies will be added as new drugs emerge against different cancers. Participation in the trial will depend on how long the benefit lasts. Type: Interventional Start Date: Jan 2020 |
Tegavivint for the Treatment of Recurrent or Refractory Solid Tumors, Including Lymphomas and Desmoid...
Children's Oncology Group
Colorectal Carcinoma
Endometrial Carcinoma
Melanoma
Neuroblastoma
Ovarian Carcinoma
This phase I/II trial evaluates the highest safe dose, side effects, and possible benefits of
tegavivint in treating patients with solid tumors that has come back (recurrent) or does not
respond to treatment (refractory). Tegavivint interferes with the binding of beta-catenin to... expand
This phase I/II trial evaluates the highest safe dose, side effects, and possible benefits of tegavivint in treating patients with solid tumors that has come back (recurrent) or does not respond to treatment (refractory). Tegavivint interferes with the binding of beta-catenin to TBL1, which may help stop the growth of tumor cells by blocking the signals passed from one molecule to another inside a cell that tell a cell to grow. Type: Interventional Start Date: Nov 2021 |
Nivolumab in Combination With Chemo-Immunotherapy for the Treatment of Newly Diagnosed Primary Mediastinal...
National Cancer Institute (NCI)
Primary Mediastinal Large B-Cell Lymphoma
This phase III trial compares the effects of nivolumab with chemo-immunotherapy versus
chemo-immunotherapy alone in treating patients with newly diagnosed primary mediastinal
B-cell lymphoma (PMBCL). Immunotherapy with monoclonal antibodies, such as nivolumab, may
help the... expand
This phase III trial compares the effects of nivolumab with chemo-immunotherapy versus chemo-immunotherapy alone in treating patients with newly diagnosed primary mediastinal B-cell lymphoma (PMBCL). Immunotherapy with monoclonal antibodies, such as nivolumab, may help the body's immune system attack the cancer, and may interfere with the ability of cancer cells to grow and spread. Treatment for PMBCL involves chemotherapy combined with an immunotherapy called rituximab. Chemotherapy drugs work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Rituximab is a monoclonal antibody. It binds to a protein called CD20, which is found on B cells (a type of white blood cell) and some types of cancer cells. This may help the immune system kill cancer cells. Giving nivolumab with chemo-immunotherapy may help treat patients with PMBCL. Type: Interventional Start Date: Oct 2021 |
The Pediatric Acute Leukemia (PedAL) Screening Trial - A Study to Test Bone Marrow and Blood in Children...
LLS PedAL Initiative, LLC
Acute Lymphoblastic Leukemia
Acute Myeloid Leukemia
Acute Myeloid Leukemia Post Cytotoxic Therapy
Juvenile Myelomonocytic Leukemia
Mixed Phenotype Acute Leukemia
This study aims to use clinical and biological characteristics of acute leukemias to screen
for patient eligibility for available pediatric leukemia sub-trials. Testing bone marrow and
blood from patients with leukemia that has come back after treatment or is difficult to treat... expand
This study aims to use clinical and biological characteristics of acute leukemias to screen for patient eligibility for available pediatric leukemia sub-trials. Testing bone marrow and blood from patients with leukemia that has come back after treatment or is difficult to treat may provide information about the patient's leukemia that is important when deciding how to best treat it, and may help doctors find better ways to diagnose and treat leukemia in children, adolescents, and young adults. Type: Interventional Start Date: Apr 2022 |
REgiStry Of the NAtural History of recurreNt periCarditis in pEdiatric and Adult Patients
Kiniksa Pharmaceuticals (UK), Ltd.
Recurrent Pericarditis
The registry will focus on furthering the understanding of the natural history of recurrent
pericarditis (RP), as well as document RP-related clinical, health-related quality of life
(HRQoL), and economic burden and will assist the medical community to refine or develop
data-driven... expand
The registry will focus on furthering the understanding of the natural history of recurrent pericarditis (RP), as well as document RP-related clinical, health-related quality of life (HRQoL), and economic burden and will assist the medical community to refine or develop data-driven recommendations for clinical management of RP patients to optimize clinical outcomes. It also aims to generate data in support of the impact of rilonacept on clinical outcomes in a real-world population. Type: Observational [Patient Registry] Start Date: Mar 2021 |
A Study of a New Way to Treat Children and Young Adults With a Brain Tumor Called NGGCT
Children's Oncology Group
Central Nervous System Nongerminomatous Germ Cell Tumor
Choriocarcinoma
Embryonal Carcinoma
Immature Teratoma
Malignant Teratoma
This phase II trial studies the best approach to combine chemotherapy and radiation therapy
(RT) based on the patient's response to induction chemotherapy in patients with
non-germinomatous germ cell tumors (NGGCT) that have not spread to other parts of the brain
or body (localized).... expand
This phase II trial studies the best approach to combine chemotherapy and radiation therapy (RT) based on the patient's response to induction chemotherapy in patients with non-germinomatous germ cell tumors (NGGCT) that have not spread to other parts of the brain or body (localized). This study has 2 goals: 1) optimizing radiation for patients who respond well to induction chemotherapy to diminish spinal cord relapses, 2) utilizing higher dose chemotherapy followed by conventional RT in patients who did not respond to induction chemotherapy. Chemotherapy drugs, such as carboplatin, etoposide, ifosfamide, and thiotepa, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Radiation therapy uses high energy x-rays or high-energy protons to kill tumor cells and shrink tumors. Studies have shown that patients with newly-diagnosed localized NGGCT, whose disease responds well to chemotherapy before receiving radiation therapy, are more likely to be free of the disease for a longer time than are patients for whom the chemotherapy does not efficiently eliminate or reduce the size of the tumor. The purpose of this study is to see how well the tumors respond to induction chemotherapy to decide what treatment to give next. Some patients will be given RT to the spine and a portion of the brain. Others will be given high dose chemotherapy and a stem cell transplant before RT to the whole brain and spine. Giving treatment based on the response to induction chemotherapy may lower the side effects of radiation in some patients and adjust the therapy to a more efficient one for other patients with localized NGGCT. Type: Interventional Start Date: Jul 2021 |
Inotuzumab Ozogamicin and Post-Induction Chemotherapy in Treating Patients With High-Risk B-ALL, Mixed...
Children's Oncology Group
B Acute Lymphoblastic Leukemia
B Lymphoblastic Lymphoma
Central Nervous System Leukemia
Mixed Phenotype Acute Leukemia
Testicular Leukemia
This phase III trial studies whether inotuzumab ozogamicin added to post-induction
chemotherapy for patients with High-Risk B-cell Acute Lymphoblastic Leukemia (B-ALL) improves
outcomes. This trial also studies the outcomes of patients with mixed phenotype acute
leukemia (MPAL),... expand
This phase III trial studies whether inotuzumab ozogamicin added to post-induction chemotherapy for patients with High-Risk B-cell Acute Lymphoblastic Leukemia (B-ALL) improves outcomes. This trial also studies the outcomes of patients with mixed phenotype acute leukemia (MPAL), and B-lymphoblastic lymphoma (B-LLy) when treated with ALL therapy without inotuzumab ozogamicin. Inotuzumab ozogamicin is a monoclonal antibody, called inotuzumab, linked to a type of chemotherapy called calicheamicin. Inotuzumab attaches to cancer cells in a targeted way and delivers calicheamicin to kill them. Other drugs used in the chemotherapy regimen, such as cyclophosphamide, cytarabine, dexamethasone, doxorubicin, daunorubicin, methotrexate, leucovorin, mercaptopurine, prednisone, thioguanine, vincristine, and pegaspargase or calaspargase pegol work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. This trial will also study the outcomes of patients with mixed phenotype acute leukemia (MPAL) and disseminated B lymphoblastic lymphoma (B-LLy) when treated with high-risk ALL chemotherapy. The overall goal of this study is to understand if adding inotuzumab ozogamicin to standard of care chemotherapy maintains or improves outcomes in High Risk B-cell Acute Lymphoblastic Leukemia (HR B-ALL). The first part of the study includes the first two phases of therapy: Induction and Consolidation. This part will collect information on the leukemia, as well as the effects of the initial treatment, to classify patients into post-consolidation treatment groups. On the second part of this study, patients with HR B-ALL will receive the remainder of the chemotherapy cycles (interim maintenance I, delayed intensification, interim maintenance II, maintenance), with some patients randomized to receive inotuzumab. The patients that receive inotuzumab will not receive part of delayed intensification. Other aims of this study include investigating whether treating both males and females with the same duration of chemotherapy maintains outcomes for males who have previously been treated for an additional year compared to girls, as well as to evaluate the best ways to help patients adhere to oral chemotherapy regimens. Finally, this study will be the first to track the outcomes of subjects with disseminated B-cell Lymphoblastic Leukemia (B-LLy) or Mixed Phenotype Acute Leukemia (MPAL) when treated with B-ALL chemotherapy. Type: Interventional Start Date: Oct 2019 |
Stopping Tyrosine Kinase Inhibitors in Affecting Treatment-Free Remission in Patients With Chronic Phase...
Children's Oncology Group
Chronic Phase Chronic Myeloid Leukemia, BCR-ABL1 Positive
This phase II trial studies how stopping tyrosine kinase inhibitors will affect
treatment-free remission in patients with chronic myeloid leukemia in chronic phase. When the
level of disease is very low, it's called molecular remission. TKIs are a type of medication
that help... expand
This phase II trial studies how stopping tyrosine kinase inhibitors will affect treatment-free remission in patients with chronic myeloid leukemia in chronic phase. When the level of disease is very low, it's called molecular remission. TKIs are a type of medication that help keep this level low. However, after being in molecular remission for a specific amount of time, it may not be necessary to take tyrosine kinase inhibitors. It is not yet known whether stopping tyrosine kinase inhibitors will help patients with chronic myeloid leukemia in chronic phase continue or re-achieve molecular remission. Type: Interventional Start Date: Nov 2019 |
Inotuzumab Ozogamicin in Treating Younger Patients With B-Lymphoblastic Lymphoma or Relapsed or Refractory...
Children's Oncology Group
Recurrent B Acute Lymphoblastic Leukemia
Recurrent B Lymphoblastic Lymphoma
Refractory B Acute Lymphoblastic Leukemia
Refractory B Lymphoblastic Lymphoma
This phase II trial studies how well inotuzumab ozogamicin works in treating younger patients
with B-lymphoblastic lymphoma or CD22 positive B acute lymphoblastic leukemia that has come
back (relapsed) or does not respond to treatment (refractory). Inotuzumab ozogamicin is a... expand
This phase II trial studies how well inotuzumab ozogamicin works in treating younger patients with B-lymphoblastic lymphoma or CD22 positive B acute lymphoblastic leukemia that has come back (relapsed) or does not respond to treatment (refractory). Inotuzumab ozogamicin is a monoclonal antibody, called inotuzumab, linked to a toxic agent called ozogamicin. Inotuzumab attaches to CD22 positive cancer cells in a targeted way and delivers ozogamicin to kill them. Type: Interventional Start Date: Jun 2017 |
Clinical and Laboratory Study of Methylmalonic Acidemia
National Human Genome Research Institute (NHGRI)
Organic Acidemia
Methylmalonic Acidemia
Inborn Errors of Metabolism
Methylmalonic acidemia (MMA), one of the most common inborn errors of organic acid
metabolism, is heterogeneous in etiology and clinical manifestations. Affected patients with
cblA, cblB and mut classes of MMA are medically fragile and can suffer from complications
such as... expand
Methylmalonic acidemia (MMA), one of the most common inborn errors of organic acid metabolism, is heterogeneous in etiology and clinical manifestations. Affected patients with cblA, cblB and mut classes of MMA are medically fragile and can suffer from complications such as metabolic stroke or infarction of the basal ganglia, pancreatitis, end stage renal failure, growth impairment, osteoporosis, and developmental delay. The frequency of these complications and their precipitants remain undefined. Furthermore, current treatment protocol outcomes have continued to demonstrate substantial morbidity and mortality in the patient population. Increasingly, solid organ transplantation (liver, and/or kidney) has been used to treat patients. Disordered transport and intracellular metabolism of vitamin B12 produces a distinct group of disorders that feature methylmalonic acidemia as well as (hyper)homocysteinemia. These conditions are named after the corresponding cellular complementation class (cblC, cblD, cblF, cblJ and cblX) and are also heterogenous, clinically and biochemically. The genetic disorders underlying cblE and cblG feature an isolated impairment of the activity of methionine synthase, a critical enzyme involved in the conversion of homocysteine to methionine and these disorders feature (hyper)homocysteinemia. Lastly, a group of patients can have increased methylmalonic acid and/or homocysteine in the blood or urine caused by variant(s)in recently identified (ACSF3) and unknown genes. In this protocol, we will clinically evaluate patients with methylmalonic acidemia and cobalamin metabolic defects. Routine inpatient admissions will last up to 4-5 days and involve urine collection, blood drawing, ophthalmological examination, radiological procedures, MRI/MRS, skin biopsies in some, and developmental testing. In a subset of patients who have or will receive renal, hepato- or hepato-renal transplants or have an unusual variant or clinical course and have MMA, a lumbar puncture to examine CSF metabolites will be performed. In this small group of patients, CSF metabolite monitoring may be used to adjust therapy. The study objectives will be to further delineate the spectrum of phenotypes and characterize the natural history of these enzymopathies, query for genotype/enzymatic/phenotype correlations, search for new genetic causes of methylmalonic acidemia and/or homocysteinemia, identify new disease biomarkers and define clinical outcome parameters for future clinical trials. The population will consist of participants previously evaluated at NIH, physician referrals, and families directed to the study from clinicaltrials.gov as well as the Organic Acidemia Association, Homocystinuria Network America and other national and international support groups. Most participants will be evaluated only at the NIH Clinical Center. However, if the NIH team decides that a patient under the age of 2 years is a candidate subject for this research protocol, that patient may enroll at the Children s National Medical Center (CNMC) site, pending approval by Dr Chapman, the Principal Investigator of the CNMC location Individuals may also enroll in the tissue collection only part of the study at the UPMC Children s Hospital of Pittsburgh or share medical history and clinical data via telemedicine visits remotely. Outcome measures will largely be descriptive and encompass correlations between clinical, biochemical and molecular parameters. Type: Observational Start Date: Jun 2004 |
Pediatric Percutaneous Ultrasound Gastrostomy Technique
CoapTech
Gastrostomy
Gastrostomy Complications
Pediatric Disorder
Ultrasound
The purpose of this research study is to test a new device called the PUMA-G Pediatric
System. The research will measure if the device works well to safely aid doctors placing
gastrostomy feeding tubes in children. The PUMA-G Pediatric System is an investigational
device that... expand
The purpose of this research study is to test a new device called the PUMA-G Pediatric System. The research will measure if the device works well to safely aid doctors placing gastrostomy feeding tubes in children. The PUMA-G Pediatric System is an investigational device that uses ultrasound and magnets to guide insertion of a feeding tube. Type: Interventional Start Date: Jul 2023 |
Use Of A Response-Adapted Ruxolitinib-Containing Regimen For The Treatment Of Hemophagocytic Lymphohistiocytosis
St. Jude Children's Research Hospital
Hemophagocytic Lymphohistiocytosis
This study is a multi-site Phase Ib/II, 2-arm non-randomized clinical trial to determine the
efficacy and tolerability of a response-adapted regimen combining ruxolitinib, dexamethasone,
and etoposide as Frontline therapy for patients with newly diagnosed hemophagocytic
lymphohistiocytosis... expand
This study is a multi-site Phase Ib/II, 2-arm non-randomized clinical trial to determine the efficacy and tolerability of a response-adapted regimen combining ruxolitinib, dexamethasone, and etoposide as Frontline therapy for patients with newly diagnosed hemophagocytic lymphohistiocytosis (HLH) or as Salvage therapy for patients with relapsed/refractory HLH. Primary Objective - To determine the efficacy and tolerability of a response-adapted ruxolitinib-containing regimen for patients with newly diagnosed HLH. Secondary Objectives - To describe the efficacy and tolerability of a response-adapted ruxolitinib-containing regimen for patients with relapsed/refractory HLH. - To describe the overall response and outcome for patients with newly diagnosed or relapsed/refractory HLH who are treated with this response-adapted ruxolitinib-containing regimen. Exploratory Objectives - To estimate the pharmacokinetic (PK) parameters of ruxolitinib, assess covariates of ruxolitinib pharmacokinetics, and test whether the drug's effectiveness is correlated with systemic drug exposure. - To query specific immunologic biomarkers and determine whether the levels of these biomarkers correlate with disease response and outcome. Type: Interventional Start Date: Jul 2021 |
Study of Efficacy and Safety of Tisagenlecleucel in HR B-ALL EOC MRD Positive Patients
Novartis Pharmaceuticals
B-Cell Acute Lymphoblastic Leukemia
This is a single arm, open-label, multi-center, phase II study to determine the efficacy and
safety of tisagenlecleucel in de novo HR pediatric and young adult B-ALL patients who
received first-line treatment and are EOC MRD positive. The study will have the following
sequential... expand
This is a single arm, open-label, multi-center, phase II study to determine the efficacy and safety of tisagenlecleucel in de novo HR pediatric and young adult B-ALL patients who received first-line treatment and are EOC MRD positive. The study will have the following sequential phases: screening, pre-treatment, treatment & follow-up, and survival. After tisagenlecleucel infusion, patient will have assessments performed more frequently in the first month and then at Day 29, then every 3 months for the first year, every 6 months for the second year, then yearly until the end of the study. Efficacy and safety will be assessed at study visits and as clinically indicated throughout the study. The study is expected to end in approximately 8 years after first patient first treatment (FPFT). A post-study long term follow-up safety will continue under a separate protocol per health authority guidelines. Type: Interventional Start Date: Jun 2019 |
International Rare Brain Tumor Registry
Children's National Research Institute
Astroblastoma
BCOR ITD Sarcoma
CNS Sarcoma
Unclassified Tumor, Malignant
The objective of the International Rare Brain Tumor Registry (IRBTR) is to better understand
rare brain tumors through the collection of biospecimens and matched clinical data of
children, adolescents, and young adult patients diagnosed with rare brain tumors.
expand
The objective of the International Rare Brain Tumor Registry (IRBTR) is to better understand rare brain tumors through the collection of biospecimens and matched clinical data of children, adolescents, and young adult patients diagnosed with rare brain tumors. Type: Observational [Patient Registry] Start Date: Jan 2023 |
Individualized Treatment Plan in Children and Young Adults With Relapsed Medulloblastoma
University of California, San Francisco
Medulloblastoma
Medulloblastoma, Childhood
Medulloblastoma Recurrent
The current study will use a new treatment approach based on the molecular characteristics of
each participant's tumor. The study will test the feasibility of performing real-time drug
screening on tissue taken during surgery, and of having a specialized tumor board assign a... expand
The current study will use a new treatment approach based on the molecular characteristics of each participant's tumor. The study will test the feasibility of performing real-time drug screening on tissue taken during surgery, and of having a specialized tumor board assign a treatment plan based on the results of this screening and genomic sequencing. The aim of this trial is to allow every child and young adult with medulloblastoma to receive the most effective and least toxic therapies currently available, and will pave the way for improved understanding and treatment of these tumors in the future. Type: Interventional Start Date: Feb 2022 |
Orphan Europe Carbaglu® Surveillance Protocol
Nicholas Ah Mew
N-acetylglutamate Synthase (NAGS) Deficiency
The purpose of this study is to conduct post-marketing surveillance of carglumic acid
(Carbaglu) to obtain long-term clinical safety information. Carglumic acid was approved by
the United States Food and Drug Administration (FDA) for treatment of acute hyperammonemia
due to... expand
The purpose of this study is to conduct post-marketing surveillance of carglumic acid (Carbaglu) to obtain long-term clinical safety information. Carglumic acid was approved by the United States Food and Drug Administration (FDA) for treatment of acute hyperammonemia due to N-acetylglutamate synthase (NAGS) deficiency. Much of the FDA-required data is already collected through the Longitudinal Study of Urea Cycle Disorders (RDCRN Protocol #5101). This study will collect additional data on adverse events (interim events), adverse reactions, pregnancy, and fetal outcomes. Type: Observational [Patient Registry] Start Date: Apr 2012 |
A Study to See if Memantine Protects the Brain During Radiation Therapy Treatment for Primary Central...
Children's Oncology Group
Central Nervous System Carcinoma
This phase III trial compares memantine to usual treatment in treating patients with primary
central nervous system tumors. Memantine may block receptors (parts of nerve cells) in the
brain known to contribute to a decline in cognitive function. Giving memantine may make a... expand
This phase III trial compares memantine to usual treatment in treating patients with primary central nervous system tumors. Memantine may block receptors (parts of nerve cells) in the brain known to contribute to a decline in cognitive function. Giving memantine may make a difference in cognitive function (attention, memory, or other thought processes) in children and adolescents receiving brain radiation therapy to treat a primary central nervous system tumors. Type: Interventional Start Date: Nov 2021 |
Enasidenib for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia Patients With an IDH2 Mutation
Children's Oncology Group
Recurrent Acute Myeloid Leukemia
Refractory Acute Myeloid Leukemia
This trial studies the side effects of enasidenib and to see how well it works in treating
patients with acute myeloid leukemia that has come back after treatment (relapsed) or has
been difficult to treat with chemotherapy (refractory). Patients must also have a specific
genetic... expand
This trial studies the side effects of enasidenib and to see how well it works in treating patients with acute myeloid leukemia that has come back after treatment (relapsed) or has been difficult to treat with chemotherapy (refractory). Patients must also have a specific genetic change, also called a mutation, in a protein called IDH2. Enasidenib may stop the growth of cancer cells by blocking the mutated IDH2 protein, which is needed for cell growth. Type: Interventional Start Date: Aug 2023 |
The Myelin Disorders Biorepository Project
Children's Hospital of Philadelphia
Leukodystrophy
White Matter Disease
Leukoencephalopathies
4H Syndrome
Adrenoleukodystrophy
The Myelin Disorders Biorepository Project (MDBP) seeks to collect and analyze clinical data
and biological samples from leukodystrophy patients worldwide to support ongoing and future
research projects. The MDBP is one of the world's largest leukodystrophy biorepositories,... expand
The Myelin Disorders Biorepository Project (MDBP) seeks to collect and analyze clinical data and biological samples from leukodystrophy patients worldwide to support ongoing and future research projects. The MDBP is one of the world's largest leukodystrophy biorepositories, having enrolled nearly 2,000 affected individuals since it was launched over a decade ago. Researchers working in the biorepository hope to use these materials to uncover new genetic etiologies for various leukodystrophies, develop biomarkers for use in future clinical trials, and better understand the natural history of these disorders. The knowledge gained from these efforts may help improve the diagnostic tools and treatment options available to patients in the future. Type: Observational [Patient Registry] Start Date: Dec 2016 |
Childhood Cancer Survivor Study
St. Jude Children's Research Hospital
Cancer
The Childhood Cancer Survivor Study (CCSS) will investigate the long-term effects of cancer
and its associated therapies. A retrospective cohort study will be conducted through a
multi-institutional collaboration, which will involve the identification and active follow-up... expand
The Childhood Cancer Survivor Study (CCSS) will investigate the long-term effects of cancer and its associated therapies. A retrospective cohort study will be conducted through a multi-institutional collaboration, which will involve the identification and active follow-up of a cohort of approximately 50,000 survivors of cancer, diagnosed before 21 years of age, between 1970 and 1999 and 10,000 sibling controls. This project will study children and young adults exposed to specific therapeutic modalities, including radiation, chemotherapy, and/or surgery, who are at increased risk of late-occurring adverse health outcomes. A group of sibling controls will be identified and data collected for comparison purposes. Type: Observational Start Date: Jan 1995 |
Azithromycin Treatment for Respiratory Syncytial Virus-induced Respiratory Failure in Children
University of Alabama at Birmingham
Respiratory Syncytial Virus Infections
The overarching hypothesis of the ARRC trial is that administration of Azithromycin (AZM)
during acute, Respiratory Syncytial Virus (RSV)-induced respiratory failure will be
beneficial, mediated through the matrix metalloproteinase (MMP)-9 pathway.
expand
The overarching hypothesis of the ARRC trial is that administration of Azithromycin (AZM) during acute, Respiratory Syncytial Virus (RSV)-induced respiratory failure will be beneficial, mediated through the matrix metalloproteinase (MMP)-9 pathway. Type: Interventional Start Date: Feb 2022 |
Volitinib in Treating Patients With Recurrent or Refractory Primary CNS Tumors
National Cancer Institute (NCI)
Recurrent Diffuse Intrinsic Pontine Glioma
Recurrent Malignant Glioma
Recurrent Medulloblastoma
Recurrent Primary Central Nervous System Neoplasm
Refractory Diffuse Intrinsic Pontine Glioma
This phase I trial studies the side effects and best dose of volitinib in treating patients
with primary central nervous system (CNS) tumors that have come back (recurrent) or does not
respond to treatment (refractory). Volitinib may stop the growth of tumor cells by blocking... expand
This phase I trial studies the side effects and best dose of volitinib in treating patients with primary central nervous system (CNS) tumors that have come back (recurrent) or does not respond to treatment (refractory). Volitinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Type: Interventional Start Date: Nov 2018 |
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